Genome editing-mediated knock-in of therapeutic genes ameliorates the disease phenotype in a model of hemophilia

• Main Authors: Jeong Hyeon Lee (Author), Hye-Kyung Oh (Author), Beom Seok Choi (Author), Ho Hyeon Lee (Author), Kyu Jun Lee (Author), Un Gi Kim (Author), Jina Lee (Author), Hyerim Lee (Author), Geon Seong Lee (Author), Se Jun Ahn (Author), Jeong Pil Han (Author), Seokjoong Kim (Author), Su Cheong Yeom (Author), Dong Woo Song (Author)
• Formato: Libro
• Publicado: Elsevier, 2022-09-01T00:00:00Z.
• Subjects: article
• Acceso en liña: Connect to this object online.

Recently, clinical trials of adeno-associated virus-mediated replacement therapy have suggested long-term therapeutic effects for several genetic diseases of the liver, including hemophilia. However, there remain concerns regarding decreased therapeutic effects when the liver is regenerated or when...